NCT07218887 · Alexion Pharmaceuticals, Inc.
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
(DCMRestore)
What this study is about
This Phase 1/2 study is an where both patients and doctors know the treatment given, dose finding and dose expansion study investigating the safety, tolerability, and effectiveness of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
View original scientific description
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Pathogenic or likely pathogenic mutation in BAG3
- Medical history of diagnosis of DCM
- Stable combination of HF SoC medications
- Adequate acoustic windows for echocardiography
Exclusion criteria
- Presence of antibodies to AAV9
- Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM.
- Decompensated HF Note: Other inclusion and exclusion criteria may apply.
Where
- Birmingham, Alabama
- Boston, Massachusetts
- Cincinnati, Ohio
- Portland, Oregon
- Philadelphia, Pennsylvania
- Houston, Texas
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 15, 2026 · Source of record for eligibility and locations